Why the study?
Guidelines conditionally recommend long-term prophylaxis for severe, frequent bleeding in von Willebrand disease, prompting investigation into whether recombinant von Willebrand factor prophylaxis is efficacious and safe.
Does recombinant von Willebrand factor prophylaxis reduce treated spontaneous bleeding events in adults with severe von Willebrand disease?
Population
Adults with severe VWD and bleeding events requiring VWF therapy in the past year
Comparison
rVWF prophylaxis in prior on-demand vs prior pdVWF switch groups compared to historical rates
Design
Prospective open-label nonrandomized phase 3 study
Follow-up
12 months
Authors
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rVWF prophylaxis may reduce spontaneous bleeds in severe VWD; leaves open need for randomized trials before practice change.
Does recombinant von Willebrand factor prophylaxis reduce treated spontaneous bleeding events in adults with severe von Willebrand disease?
Recombinant von Willebrand factor prophylaxis effectively reduces treated spontaneous bleeding events in patients with severe von Willebrand disease previously on on-demand therapy and maintains hemostatic control in those switching from plasma-derived VWF.
Leebeek et al. (2022) studied this question.
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